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Phase 2 Study of Apitegromab in Patients with Facioscapulohumeral Muscular Dystrophy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

Facioscapulohumeral Muscular Dystrophy is a rare genetic condition that leads to gradual weakness of the muscles of the face, shoulder blades, and upper arms. The study will compare an experimental medicine called apitegromab with a simple saline solution (sodium chloride) that looks the same but does not contain the active drug. Both are given by an intravenous infusion, which means the medication is delivered directly into a vein through a small tube.

The purpose of the study is to find out if apitegromab can improve muscle health compared with the control. Participants will receive a series of infusions over about a year and will have regular check‑ups that include full‑body MRI scans—detailed pictures of the whole body made with a special scanner—to see how muscle size and the amount of fat that has built up in the muscles change over time. Blood tests and safety examinations will also be done, and changes will be recorded at the start, at 24 weeks, and at 52 weeks.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization and Start of Treatment

    You will be randomly assigned to receive either the study drug apitegromab or a placebo solution.

    The assignment is double‑blind, meaning neither you nor the study staff will know which product you receive.

  2. Step 2

    Baseline Assessments

    Before the first infusion, a full‑body MRI scan will be performed to measure muscle volume.

    Blood samples will be taken to check laboratory values, vital signs will be recorded, and an electrocardiogram (ECG) will be done to assess heart health.

  3. Step 3

    First Intravenous Infusion

    You will receive an intravenous infusion of the assigned product.

    If you are assigned to apitegromab, the dose is 0000 mg per kilogram of body weight.

    If you receive the placebo, the infusion contains 000 mg per millilitre of sodium chloride solution.

  4. Step 4

    Ongoing Infusion Visits

    You will continue to receive the same type of infusion at regular visits throughout the 52‑week study period.

    The exact schedule of visits will be arranged by the study team, and each infusion will be administered intravenously.

  5. Step 5

    Mid‑Study Assessments at Week 24

    At week 24, a second full‑body MRI will be performed to evaluate changes in muscle volume and fat content.

    Additional blood tests will be taken to measure the concentration of apitegromab (if you are receiving it) and other muscle‑related markers.

    Safety checks, including vital signs and ECG, will be repeated.

  6. Step 6

    Final Assessments at Week 52

    At the end of the 52‑week period, a final full‑body MRI will be conducted to assess overall changes in muscle tissue.

    Blood samples will be collected again to evaluate serum levels of apitegromab and other study markers.

    Comprehensive safety evaluations, including laboratory tests, vital signs, and ECG, will be completed.

  7. Step 7

    Safety Monitoring Throughout the Study

    You will be asked to report any adverse events or health changes at each visit.

    The study team will monitor your safety continuously and may perform additional tests if needed.

Who can join the trial?

6 criteria

  • Be able to understand the study information and sign a written permission form called informed consent before any study activities.
  • Be willing and able to follow all study rules, attend all visits, and complete all tests that are part of the study.
  • Be a man or woman who is between 18 and 60 years old when signing the consent form.
  • Have a confirmed genetic test showing either FSHD1 or FSHD2, the two known forms of Facioscapulohumeral Muscular Dystrophy, with paperwork from an approved laboratory.
  • Have a disease severity score between 1.5 and 3.0 on the Ricci score (a scale that ranges from 0 to 5) at the screening visit.
  • Be able to walk 10 meters in 5 seconds or less on the 10‑meter walk test (10MWRT) at the start of the study.

Who cannot join the trial?

12 criteria

  • Having any other health problem, a lab test result, or an ECG reading that the doctor thinks could be unsafe, make it hard to follow the study rules, stop you from finishing the study, or affect the study’s results.
  • Pregnant or breastfeeding (carrying a baby or feeding a baby with breast milk).
  • A past history of alcoholism or use of illegal drugs (drugs that are not prescribed).
  • Any reason you cannot safely have an MRI scan, such as certain implanted electronic devices, cochlear implants, metal objects in the body, metal clips on blood vessels, metal implants, severe fear of enclosed spaces (claustrophobia), allergy to the contrast dye used in the scan, inability to stay still, or medical devices that cannot be removed.
  • Previous severe allergic reaction (hypersensitivity) to a type of medication called a monoclonal antibody (mAb) or to the study drug apitegromab or its inactive ingredients (excipients).
  • Any serious illness or condition that affected your health within the 7 days before screening, such as a current infection that spreads through the whole body (systemic infection), needing urgent treatment, or being in the hospital for any reason.
  • Having taken apitegromab before, or any other medicines that block the protein myostatin (called anti‑myostatin therapies, including drugs that block the activin receptor).
  • Using or having used muscle‑affecting substances such as anabolic steroids, growth hormones, or drugs that act on the glucagon‑like peptide‑1 receptor (a type of diabetes medication).
  • Using medicines or treatments that can strongly affect muscles (for example, androgen hormones, insulin‑like growth factor, growth hormone, systemic beta‑agonists, botulinum toxin, muscle relaxants, or supplements meant to boost muscle) or nerves that control muscles (such as acetylcholinesterase inhibitors) within 60 days before screening.
  • Taking systemic (whole‑body) steroids or corticosteroids within 60 days before screening. (Inhaled or skin‑applied steroids are allowed.)
  • Taking medicines that interfere with blood clotting (coagulation) or platelet function, or having a history or current problem with blood clotting (coagulopathy).
  • Being in another clinical study with an investigational drug within the past 3 months or within 5 half‑lives of that drug (the time it takes for half of the drug to leave the body), whichever is longer.
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Investigated drugs

APITEGROMAB is an investigational medicine being tested to see if it can help people with facioscapulohumeral muscular dystrophy (FSHD). In this study, participants receive the drug through an IV infusion, which means it is given directly into a vein. The purpose of giving APITEGROMAB is to evaluate whether it can improve muscle strength and function compared with a placebo. Researchers will monitor participants for safety and any side effects while also measuring how well the drug works over the 52‑week trial period. The drug is considered an orphan drug, meaning it is intended for a rare disease like FSHD.

What is already known about the treatment

  • Isotonische Natriumchlorid-Lösung 0,9 % DELTAMEDICA Infusionslösung

    This medication is given as an intravenous solution that is infused directly into a vein. It is a widely used, approved isotonic saline that helps keep the body’s fluid and salt balance normal, and it is often used as a placebo in clinical studies. The solution works by supplying sodium and chloride ions, which restore the normal concentration of fluids outside cells. It is classified as an electrolyte solution and is listed under the ATC code B05CB01.

  • APITEGROMAB

    Apitegromab is administered by intravenous infusion, where the drug is slowly injected into a vein. It is an investigational monoclonal antibody that is currently being studied in clinical trials for the rare muscle disease facioscapulohumeral muscular dystrophy. The antibody works by blocking a protein called myostatin, which normally limits muscle growth, thereby helping muscles become stronger. It belongs to the class of myostatin‑inhibiting antibodies and is designated as an orphan drug.

Investigated diseases

Facioscapulohumeral Muscular Dystrophy - It is a genetic disorder that causes the muscles of the face, shoulder blades, and upper arms to become weak. Symptoms often start in teenage years or early adulthood, with difficulty raising the arms or smiling. The weakness spreads slowly to other muscles such as the trunk, hips, and lower limbs. Over time, the affected muscle tissue is gradually replaced by fat, which can be seen on imaging scans. The condition usually progresses slowly, leading to a gradual loss of muscle strength over many years.
Trial detailsLast updated 7 Nov 2026
Age18+ yearsPhasePhase IITrial ID2026-525204-10-00Protocol codeSRK-015-009Estimated enrolment48 patientsSponsorScholar Rock Inc.

sourced from the EU Clinical Trials Register and site verification

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